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Startup Tests Drugs Aimed at Autism 171

Posted by kdawson
from the whose-x-you-callin'-fragile dept.
An anonymous reader sends in this link from Technology Review about a startup company testing drugs that may help those with autism-spectrum disorders — even adults. "Seaside Therapeutics, a startup based in Cambridge, MA, is testing two compounds for the treatment of fragile X syndrome, a rare, inherited form of intellectual disability linked to autism. The treatments have emerged from molecular studies of animal models that mirror the genetic mutations seen in humans. Researchers hope that the drugs, which are designed to correct abnormalities at the connections between neurons, will ultimately prove effective in other forms of autism spectrum disorders. ... The company is funded almost entirely by an undisclosed family investment of $60 million, with $6 million from the National Institutes of Health. [A spokesman] says that Seaside has enough funding to take its compounds through clinical testing and approval."
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Startup Tests Drugs Aimed at Autism

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  • Re:Do I have it (Score:4, Informative)

    by MichaelSmith (789609) on Monday January 11, 2010 @05:56AM (#30721150) Homepage Journal

    If you were really autistic you would lurk and never post.

  • $60m is pocket-money (Score:3, Informative)

    by PDoc (841773) on Monday January 11, 2010 @06:16AM (#30721202) Homepage
    Seriously, $60m isn't anywhere near enough to bring this to market. Most studies in pharma show that $1000m is far closer to the real figure these days, with some pushing that towards $1700m [drugresearcher.com]. Of course, this is an average figure, and the costs of drug development are highest towards the end (phase IIb, phase III). Any drug targeting the CNS is going to be expensive in trials, and with the condition apparently 'rare' (an ill-defined term), finding suitable patients willing to undergo the treatment in trials might be difficult. More realistically, $60m might get them to the point where a Big-Pharma will either buy the company or the drug.
  • Re:Do I have it (Score:5, Informative)

    by DangerFace (1315417) on Monday January 11, 2010 @06:32AM (#30721250) Journal

    Then again, if you had fragile X syndrome you wouldn't actually have autism. This is a deeply misleading article title and summary, since Autistic Spectrum Disorder (ASD) covers a wide range of psychological profiles and is deeply misunderstood by most people - even plenty of people who work with it every day. You will notice, reading the article (yeah, I must be new here) that none of the scientists mention ASD. The guy who wrote this piece just thought that would give him an angle, since no one has heard of fragile X syndrome, but everyone loves a good autism story, despite (because of?) most people never having met someone with serious levels of ASD.

    Just to clear things up, fragile X syndrome is a chromosomal abnormality that causes various physical deformities and some forms of mental retardation. This [wikipedia.org] is acceptable of you want to know more. There is some limited evidence that correlation exists between some forms of ASD and fragile X syndrome, but causality is far from demonstrated.

    Additionally, ASD is defined as being a "pervasive developmental disorder", meaning that a) symtoms must be present from fairly early on in life and b) autism is an innate part of the person suffering from it, and a cure not only doesn't exist - the concept of a cure is nonsensical. Don't get me wrong, I would love there to be a cure for ASD, but medical science currently defines it as uncurable. As an analogy, it would be like trying to 'cure' someone of having social function and being capable of imaginitive play - you could teach them limited functions to appear like they had no grasp of the abstract, but you couldn't turn them autistic.

    The media, and people in general, need to cease this endless obsession with autism - it's an incredibly complex subject, and studying it for years only allows you to scratch the surface (trust me on this). Being crap with people suggests some form of social, behavioural, or anxiety disorder. ASD is a serious disorder with serious consequences. Rainman does not exist. As a rule of thumb, if you can put together a fully formed sentence, you almost certainly don't have meaningful levels of ASD. If you can read facial expressions without spending years actually consciously memorising what faces mean what, you don't have meaningful levels of ASD. Okay, if you've gotten this far you might have comparatively mild Asperger's or something on that end of the spectrum, but it'll be clinically relevant only in a small fraction of a percent of that already small group.

  • Re:Assumption much? (Score:5, Informative)

    by scapermoya (769847) on Monday January 11, 2010 @07:10AM (#30721408) Homepage
    here we go, buddy:

    the article is about a drug that targets a rare genetic trait. because the article appears in layman media and is remotely linked to autism, the submitter titled the /. story "Startup Tests Drugs Aimed at Autism," which is only mildly true.

    My original comment:

    "i sure hope autism isn't something that is more-or-less cemented at birth, making drugs like these not very useful."

    i was tacitly talking about the minority of autism cases linked to this fragile X syndrome, as evidenced by the fact that I was talking about "drugs like these." I was trying to make the point that, even though the drug has been shown to mitigate some of the symptoms of fragile X in adult animals, this tells us nothing about whether the drug will have an effect on autism. i.e. autism's link to fragile X could be completely unrelated to the symptoms of fragile X seen in the animal models (seizures, abnormal protein synthesis, etc). We have no idea what all the functions of FMRP are. anyone who says we do is a fool.

    it is entirely possible that mGluR5 has nothing to do with autism. it could simply be a receptor in a downstream pathway from FMRP, separate from whatever pathway(s) are involved with autism development. furthermore (getting back to my first post), even if this receptor is somehow involved with autism, it could be involved only at a very specific stage in development. thus, giving mGluR5 antagonists to people who have passed that stage would have no effect.

    thus your comment:
    "Whether it's cemented at "birth" is beside the point of this drug as it attempts to correct a current state not prevent one. They claim it works on adult animals they have tested."
    is practically worthless, even without the rude bit at the end that I left out. they have only shown that some non-autism symptoms of fragile X are mitigated in adult mice. it's poor form to extrapolate as you seem to be doing when there is no evidence to support it. might i recommend a biochemistry course?

    i sincerely hope these drugs do work. but even if they do it will only affect ~5% of the population of people with autism.
  • Re:What if (Score:5, Informative)

    by jonaskoelker (922170) <jonaskoelker@gnu. o r g> on Monday January 11, 2010 @07:15AM (#30721424) Homepage

    Autism seems to be on the rise and some families seem to point out patterns - pre vaccines, happy, post vaccines dolphin-esque.

    I think it would be interesting to see what would happen if everybody stopped vaccinating their kids until well after autism's typical age of onset.

    Although I think I know the answer: we would have just as many autistic kids, which would suggest that it isn't the vaccines causing the autism, yet a few people will cling to their belief no matter the evidence against it.

  • Re:What if (Score:5, Informative)

    by Paul Jakma (2677) <paul+slashdot@jakma.org> on Monday January 11, 2010 @07:31AM (#30721502) Homepage Journal

    some families seem to point out patterns - pre vaccines, happy, post vaccines dolphin-esque.

    This probably originates from a single study in the UK more than ten years ago that linked the MMR vaccine with increased incidences of autism. That study has been since been thoroughly debunked and discredited [timesonline.co.uk]. Stop repeating it.

  • Re:Do I have it (Score:2, Informative)

    by morgan_greywolf (835522) on Monday January 11, 2010 @09:40AM (#30722192) Homepage Journal

    One of the reasons that the media, and people in general, have seemingly become obsessed with autism is that there has been a very significant rise in diagnosis of autism spectrum disorders. Furthermore, autism spectrum disorders are often misdiagnosed as other problems. Finally, the public is not aware that autism spectrum disorders cover a range of different, distinct disorders, from very low functioning varieties to very high functioning varieties.

  • Re:What if (Score:3, Informative)

    by Jason Levine (196982) on Monday January 11, 2010 @10:35AM (#30722888)

    I think the bigger skew to the risk judgment is that vaccines have been too successful for their own good in some respects. Do you remember when polio paralyzed people regularly, measles killed and a cough might being fears of being Whooping? If you're around my age (34) or even a bit older, you don't. Neither do I. Of course, I've read many accounts, but haven't seen it first hand. So it would be easy to discount the threat that these diseases pose.

    Some people think: "I haven't seen anyone I know of die or be maimed by polio/measles/whooping cough, so how bad can it be?" (Yes, one antivax nut in Australia even said that whooping cough has never killed anyone!) If they had actually had a cousin die from whooping cough, had a sibling scarred for life from measles and had a friend who barely survived polio but will be in a wheelchair the rest of his life, I doubt they'd be so anti-vaccine.

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